Treatment of Polycystic Ovary Syndrome (PCOS)
Multicomponent lifestyle intervention including diet, exercise, and behavioral strategies is the first-line treatment for all women with PCOS, regardless of body weight, followed by symptom-specific pharmacological therapy based on reproductive goals. 1, 2
First-Line Treatment: Lifestyle Modification
All women with PCOS should begin with lifestyle intervention as the foundation of treatment, targeting a 30% energy deficit or 500-750 kcal/day reduction. 1, 2 This approach improves reproductive, metabolic, and psychological outcomes even in lean women with PCOS, as insulin resistance affects women regardless of BMI. 1
Components of Lifestyle Intervention:
Diet modification: No specific diet is superior, but evidence supports low glycemic index foods, high-fiber intake, omega-3 fatty acids, Mediterranean diet patterns, and anti-inflammatory foods to improve insulin sensitivity and hormonal balance. 1, 3
Exercise: Both aerobic and resistance training improve insulin sensitivity and metabolic outcomes, with benefits occurring even without weight loss. 1, 4
Behavioral strategies: Include education modules, adherence support, and mental health management, as women with PCOS have higher rates of depression, anxiety, and eating disorders. 1, 2
Weight loss target: Even modest weight loss of 5% of initial body weight significantly improves ovulation, pregnancy rates, and metabolic parameters. 2, 5
Pharmacological Treatment Based on Reproductive Goals
For Women NOT Attempting Pregnancy:
Combined oral contraceptives are first-line pharmacological treatment, as they suppress ovarian androgen secretion and increase sex hormone binding globulin, addressing hyperandrogenism and menstrual irregularity. 2
For Women Attempting Pregnancy:
Clomiphene citrate is first-line pharmacological treatment after lifestyle modification, with approximately 80% of patients ovulating and half of those conceiving. 2, 5, 6
Start clomiphene on day 5 of the cycle after properly timed coitus is established. 6
Limit treatment to 6 total cycles (including 3 ovulatory cycles) to avoid complications. 6
Critical caveat: Patients with PCOS are unusually sensitive to gonadotropins and may have exaggerated responses, requiring the lowest effective dose and shortest treatment duration for the first course. 6
If clomiphene fails, low-dose gonadotropin therapy is next-line due to lower risk of ovarian hyperstimulation syndrome. 2
Role of Metformin:
Metformin improves insulin sensitivity, reduces ovarian androgen production, and appears safe during pregnancy, though its effects on early pregnancy outcomes are not fully established. 2, 5 It reduces insulin levels and improves glucose tolerance over time. 5
Metabolic Screening and Monitoring
All women with PCOS require metabolic screening regardless of body weight, including: 2
- Fasting glucose and 2-hour glucose tolerance test
- Lipid profile
- Blood pressure monitoring
- Regular screening for type 2 diabetes and cardiovascular disease
Common pitfall: Do not neglect metabolic screening in normal-weight PCOS patients, as insulin resistance occurs independent of BMI. 2
Preconception and Pregnancy Management
Women with PCOS attempting pregnancy require preconception counseling about increased pregnancy risks and metabolic screening before conception. 5
Once pregnant, women with PCOS need: 5
- Closer monitoring throughout pregnancy
- Regular blood pressure, kidney function, and proteinuria monitoring
- Low-dose aspirin from week 12 to week 36 to reduce preeclampsia risk
Warning: Ovarian Hyperstimulation Syndrome (OHSS)
OHSS can progress rapidly within 24 hours to several days and become life-threatening. 6 Early warning signs include abdominal pain and distention, nausea, vomiting, diarrhea, and weight gain. 6 Severe cases manifest with gross ovarian enlargement, ascites, oliguria, pleural effusion, and potentially death from hypovolemic shock or thromboembolism. 6
To minimize risk: Use the lowest effective dose of clomiphene, perform pelvic examination before each treatment course, and discontinue therapy if ovarian enlargement occurs until ovaries return to pretreatment size. 6
Treatment Hierarchy Algorithm
- Initiate lifestyle modification (diet + exercise + behavioral strategies) for ALL patients 1, 2
- Perform metabolic screening (glucose, lipids, blood pressure) 2
- Assess reproductive goals:
- Consider metformin for insulin resistance and metabolic risk reduction 2, 5
- Screen for mental health disorders (depression, anxiety, eating disorders) 2
- Monitor long-term for diabetes and cardiovascular disease 2